Evolving R&D and regulatory pathways: exploiting the potential of new science and technology

Improved understanding of the genetic causes of certain diseases, including neuro-degenerative, respiratory, and cardio-vascular diseases, will help to develop new prevention and treatment pathways and identify patients that will better respond to a treatment. In combination with new sources of data, mobile technologies, and sensitive diagnostics, these advances should not only provide needed therapies to patients sooner, but also reduce the costs of development to all healthcare stakeholders. However, are the current regulatory and healthcare frameworks ready for this scientific evolution?

Science and technologies, as well as new sources of information, offer a wealth of opportunities to optimise R&D. However, it is essential that these advances are also reflected in regulatory and clinical practice to ensure that this potential is exploited – and that patients can obtain much needed prevention and treatments much faster. 

The R&D processes, as well as regulatory and HTA evaluation, healthcare decision frameworks and delivery systems evolve more slowly than science and technology. It is not always clear whether they can always enable earlier availability of safe and effective medicines, and in particular targeted medicines, in a framework that will be financially viable for all stakeholders.

The European Medicines Agency has highlighted their willingness to seek new regulatory pathways. It states, in a 2013 press release: “Medicines regulation today is characterised by the increasing complexity of applications for new medicines, such as nanomedicines or personalised medicines, and the drug-development environment as a whole… An innovative evaluation framework involving iterative phases of data gathering and regulatory evaluation is needed in order to align regulatory approval more closely with patients’ needs for timely access to innovative medicines. This also includes the ability to integrate in the decision-making process multiple data sources – not only industry studies but also data from real-world use, as well as the views of patients on the level of risk acceptable for a given medical benefit.”

In April 2013, EFPIA published an article outlining the 6 main challenges,-- issues that can be successfully addressed only with the collaboration of all relevant parties if we are to implement this vision of more closely meeting patient needs:

  • Redefine diseases by their underlying molecular mechanisms.
  • Adapt regulatory frameworks to new science.
  • Develop new trial design and statistical methods.
  • Establish agreement on benefit–risk assessment by regulatory authorities.
  • Explore new approaches for granting earlier patient access to important new medicines.
  • Ensure regulatory operational excellence.
  • Promote global medicines development.

Some of these require collaborative research projects to jointly define, develop and test new tools and methodologies, and to make sure that outcomes of the many research and policy initiatives are exploited (e.g. Innovative Medicines Initiative). Other questions call for solutions related to legal, regulatory and medical practice.  Any of these can also succeed with continued multi-stakeholder engagement and dialogue, to which EFPIA and its members are fully committed.

Ultimately, a revised regulatory framework to promote innovation in healthcare would support the translation of scientific breakthroughs from the lab to patients, increasing innovation in the life sciences and creating benefits to society. Without improvement, the barriers to providing the rapid deployment of new therapies will continue to mount, eventually putting the entire healthcare ecosystem at risk.

  • “We are losing a lot of research from Europe because of red tape... Personalised medicine promises a wealth of new possibilities for European patients, by making healthcare delivery as tailored to the individual as their fingerprint”. (Irish Health Minister James Reilly speaking at the European Alliance for Personalised Medicine (EAPM) conference on Innovation and Patient Access to Personalised Medicines)
  • Getting a medicine to patients eight years earlier than is possible under the traditional drug development cycle – with the support of payers – is not as inconceivable as some might imagine (Hans-Georg Eichler, M.D., Frank Pétavy, M.Sc., Francesco Pignatti, M.D., and Guido Rasi:Informa UK 2013, Scrip Regulatory Affairs, “New Medicines Eight Years Faster to Patients”)
  • We are heading for new forms of clinical research for Personalised Medicine and all of us – that’s pharma, academia, payers, regulators – need to move forward to a new form of collaboration.” (Denis Lacombe, Director of the European Organisation for Research and Treatment of Cancer (EORTC), EORTC & EAPM Press Release, October 23, 2013, Europe’s health policies ‘must change’ with stakeholders leaving their ‘comfort zone’)